SmartLabs’ Post

🔊 It's great news that BlueRock received FDA Regenerative Medicine Advanced Therapy designation for Parkinson’s disease cell therapy candidate #bemdaneprocel. As of March 2024, the FDA had granted 109 RMAT destinations, of which only Neurocrine and Voyager's gene therapy (VY-AADC) was for Parkinson's disease. Here are some drugs that have been approved by the FDA via RMAT designation: ◾ Gene Therapy #Lenmeldy by Orchard Therapeutics, for the treatment of children metachromatic leukodystrophy ◾ Cell Therapy #Amtagvi by Iovance Biotherapeutics, Inc. Biotherapeutics, for the treatment of adult metastatic melanoma ◾ Gene Therapy #Lyfgenia by bluebird bio for the treatment of sickle cell disease ◾ Gene Therapy #Casgevy by Vertex Pharmaceuticals for the treatment of sickle cell disease ◾ Gene Therapy #Roctavian by BioMarin Pharmaceutical Inc. for the treatment of adults with severe hemophilia A ◾ Gene Therapy #Vyjuvek by Krystal Biotech, Inc. for the treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa ◾ Tissue based therapy #Rethymic by Enzyvant Therapeutics for the treatment of Immune reconstitution in pediatric patients with congenital athymia ◾ Tissue based therapy #StrataGraft by Stratatech for the treatment of deep partial thickness burns ◾ Cell Therapy #Breyanzi from Juno Therapeutics/Bristol Myers Squibb for the treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphoma

Offering solution to our patients is paramount. 

Check out Luca Giani, CEO of Ilios Therapeutics, in our latest spotlight video! 🎥   Ilios is leading the charge against neurodegenerative diseases by developing rationally designed, multi-targeted therapeutics. Their chemistry platform combines nature's building blocks with cutting-edge science to design multi-targeted brain-penetrant small molecules that address crucial disease processes in neurodegenerative conditions like ALS and Alzheimer’s Disease.    Watch here: https://lnkd.in/eRxMMu9K #ALS #Alzheimers #aiforgood

Coya Therapeutics, Inc. (NASDAQ:COYA) CEO Dr. Howard Berman joined Steve Darling from Proactive to share news regarding the company's recent financial results and developments. Notably, Coya Therapeutics presented positive outcomes from a proof-of-concept clinical study involving low-dose interleukin-2 (ld IL-2) in patients with Amyotrophic Lateral Sclerosis (ALS) during the second quarter. The study revealed several encouraging findings, including enhanced Treg (T-regulatory cell) function during treatment, a halt in disease progression at the 24-week mark, and minimal decline at 48 weeks. Additionally, the treatment appeared to be well-tolerated by the patients. These promising results were presented at the 2023 Muscular Dystrophy Association Conference. Watch at #Proactive #ProactiveInvestors #NASDAQ #COYA http://ow.ly/wOjl104RrpB

Very interesting allogeneic cell therapy commentary from the Poseida Therapeutics, Inc. leadership. Moving forward, the key question is: How are allogeneic cell therapy companies innovating their intellectual property (IP) licensing/collaboration practices to effectively improve donor cell engineering? #immunotherapy #immunooncology #CGT https://lnkd.in/e7_tZP9g

Imagine what you could do with 40% more cells after every isolation… Make it a reality with Akadeum's Alerion Microbubble Cell Separation System and our Human T Cell Leukopak Isolation Kit. Find out how Poseida Therapeutics is benefitting from the Alerion in this Case Study: https://hubs.li/Q02FVWDc0 #morecells #akadeum #celltherapy #cellisolation

🥇Check out the Editor's Choice #Review "Ferroptosis in Hepatocellular Carcinoma: Mechanisms, Drug Targets and Approaches to Clinical Translation" 👏by Dino Bekric, Matthias Ocker et al. Paracelsus Medizinische Privatuniversität, Boehringer Ingelheim 📌Access the full paper here: https://lnkd.in/daK_BDRX Keywords: ferroptosis; iron; hepatocellular carcinoma; drug development; combinatory treatment

Do you want to learn more about RNA therapeutics and their unfolding opportunities to bring life changing therapies to patients? Join our experts on 12 October to discuss on strategies to accelerate mRNA therapies and enable scalability and process efficiency. Learn from Dr. Andrew Geall how to improve the performance of self-replicating RNA and discuss with Dr. Bastian Rouven Brückner, Conner Feldmann, and Dr. Jason Sterling how to overcome process and analytical challenges on the journey of mRNA-based drugs to market. #mRNA #mRNAtherapeutics #RNA therapeutics

Types of RNA therapies.......

Cell therapy history was made, FDA approves first two Eylea biosimilars, Gilead's $4.3B liver disease bet yields positive ph3 data, and a trilogy of lung disease positive trial data. All this and more in today's TLDR Biotech newsletter - it's the latest issue on the homepage https://tldrbio.tech/. If you don't want to miss any of these daily updates, you can sign-up to the newsletter using the above link. #biotechnews #biotech #pharmnews #pharma #tldrbiotech